National Pediatric Transplant Week is part of Donate Life America’s National Donate Life Month, which spreads awareness about organ, eye, and tissue donation. This year, April 18th
marks the week dedicated to championing the message of ending the pediatric transplant waiting list.
A transplant is an organ, tissue, or group of cells taken from one person (the donor) and transplanted into another person (the recipient) or moved from one location to another within the same person. Transplants can be used in many ways, such as for organs (heart, kidney, etc.), tissue (skin, bone, etc.), cells (bone marrow and stem cells), and limbs (hands, feet, etc.). Transplant donors are found within a national registry, known as Be The Match in the U.S. or within an international registry, such as The International Registry in Organ Donation and Transplantation (IRODaT).
Depending on the source used for transplant, some recipients may also benefit from private storage within a family bank, as is the case with cord blood and cord tissue stem cells. In light of this week’s significance, we are taking a closer look at the status of transplants in the U.S. and how donating or preserving your baby’s umbilical stem cells has the potential to save a life.
What do transplant statistics reveal?
Statistics provided by Donate Life America indicate:
- “More than 1,900 children under the age of 18 are on the national transplant waiting list.
- More than 2.5 million tissue transplants heal lives each year.”
Additionally, a proclamation issued by the White House regarding the importance of the Donate Life America organization’s movement highlighted the following statistics:
- “Nearly 18,000 people are diagnosed each year with illnesses for which blood stem cell transplantation — requiring marrow or cord blood — is the best treatment option.
- Every day, 17 people in America die while waiting for a transplant.”
Last year’s statistics given by the Health Resources & Services Administration (HRSA) related particularly to pediatric transplants reveal:
- “Over 1,700 children received transplants in 2020.
- There were over 860 pediatric organ donors in 2020. Although they ranged from newborns to age 17, most were age 11 to 17.”
Why are umbilical cord-derived stem cells used in transplantation?
There are three sources of blood-forming cells used in hematopoietic stem cell transplants (HSCT): bone marrow, cord blood, and peripheral blood. Hematopoietic stem cells have the potential to reconstitute any blood cell type and treat children with malignancies and other types of life-threatening diseases. Determining the type of cell source to use depends on a few different factors, but cord blood can be a preferable source because:
- It is cryogenically stored and readily available when needed.
- It doesn’t need to be as closely matched to the recipient as bone marrow does.
- Studies show that transplant complications such as graft vs. host disease are less common among cord blood transplants than among marrow and peripheral blood transplants.
Learn more about the benefits
of cord blood and tissue stem cells.
According to figures given by the HRSA, “more than 300,000 cord blood units are on the donor registry [in the U.S]. This total includes over 100,000 units from the National Cord Blood Inventory (NCBI), with almost 4,000 NCBI units added in 2020.” However, finding a suitable unrelated donor within the registry can be difficult for patients of racial and ethnic minorities. Cord blood is a suitable option for patients of mixed heritage because of its ability to withstand a higher level of human leukocyte antigen (HLA) disparity. Because of cord blood’s unique qualities, cord blood transplants are theoretically possible for an estimated 95%
of pediatric patients regardless of their ethnic background.
The Cord Blood Association lists some interesting facts about cord blood transplants, such as:
- “Among unrelated hematopoietic stem cell transplants today, cord blood is the source for about 40% of pediatric patients and 10% of adult patients.
- The first cord blood transplant was performed in 1988, using matched sibling cord blood to treat a 5-year-old child with Fanconi anemia. The donor was his newborn sister. The patient is alive and well today, 30 years later.
- Cord blood donation rates vary from country to country. In the United States, where nearly 4 million babies are born each year, cord blood is collected for donation in fewer than 5% of births.”
Why should I bank my baby’s cord blood and cord tissue stem cells?
Many parents choose to store their baby’s umbilical cord blood and cord tissue stem cells with a private cord blood bank. Doing so ensures that families can have prompt and exclusive access to their specimen if needed. A baby’s own cord blood is always a 100% match for the baby, making an autologous transplant or infusion the preferred method for treating many conditions. Blood-related siblings have a 75% chance of being an acceptable match, making banking for each sibling a critical priority. Similarly, a parent is always at least a 50% match, which is considered acceptable.
The stem cells found within cord blood are currently used to treat nearly 80 diseases, including numerous types of malignancies, anemias, inherited metabolic disorders, and deficiencies of the immune system. However, cord blood and cord tissue derived stem cells are used in clinical trials to treat various other conditions. Currently, in the U.S., the FDA only allows regenerative medicine therapies as part of registered clinical trials. Many of these clinical trials involve the treatment of autism, cerebral palsy, stroke, brain injury, and other neurological conditions using umbilical stem cells.
A large majority of these trials are performed at The Marcus Center for Cellular Cures at Duke University, under the direction of Dr. Joanne Kurtzberg, who also serves as Cryo-Cell’s Medical Director. In 2017, Duke was granted an Investigational New Drug Application (IND), which allows patients to receive access to cellular therapies for several conditions through its Expanded Access Program (EAP). This program has enabled hundreds of families who have stored their children’s cord blood to take advantage of investigational therapies offered at The Marcus Center for Cellular Cures at Duke University. Children with various brain disorders, such as autism, apraxia, cerebral palsy, congenital hydrocephalus, hypoxic brain injury, and stroke, are eligible to participate.
What is the future of cord blood banking with Cryo-Cell?
Soon, families will have access to additional treatment options using their newborn’s preserved stem cells. An exclusive collaborative license agreement with Duke University grants Cryo-Cell the rights to proprietary processes and regulatory data related to cord blood and cord tissue developed at The Marcus Center for Cellular Cures at Duke. Furthermore, Cryo-Cell has been granted exclusive commercial rights to Duke’s clinical expertise and manufacturing protocols associated with various applications of cord blood and cord tissue stem and progenitor cells. The same access to treatments currently offered to patients under Duke’s FDA Expanded Access Program (EAP) will also be available within one of our anticipated clinic locations. Cryo-Cell expects to open up our first infusion clinic in January 2022.
The demand for these types of therapies continues to overwhelm the limited capacity available, with a waitlist several months long. These experimental treatments call for stringent selection criteria, including the number of cells required for a child's infusion per body weight. Since children grow quickly, it is critically important to provide access to therapy before the child outgrows the number of cells available for the treatment. Licensing of Duke's EAP treatment protocols allows Cryo-Cell to offer the same high-quality care they come to expect from an FDA-sponsored program within our clinic(s).
We recognize the demand for hematopoietic stem cell transplants, especially among pediatric patients. Our transplant/infusion record indicates that the majority of transplant recipients, using both autologous (belonging to the recipient) and allogenic (belonging to someone else, i.e., a sibling) stem cells from preserved cord blood, have been children ranging from 2 months-15 years old; with a median age of five. Our updated transplant/infusion record includes the addition of nine recent recipients that underwent transplants to treat cerebral palsy, brain injury, and, more commonly, autism using cord blood stored with Cryo-Cell. In the near future, families will have access to additional treatment opportunities using their baby’s preserved stem cells. Through our clinic's anticipated opening, we aim to reduce the waitlist time for patients presently receiving these treatments at Duke University. In turn, pediatric patients will have the best possible chance of achieving a desirable treatment outcome.
If you would like to learn more about preserving your baby’s umbilical cord-derived stem cells, please join us for an upcoming virtual seminar! You'll have the opportunity to speak with one of our cord blood educators and to take advantage of exclusive savings!